～臨床開発担当者のための PRO 利用の手引き～

1 Patient Reported Outcomes PRO 7 Ve r 1 . 0 2016 6 .. 1 1 PRO .. 2 PRO .. 2 PRO .. 3 .. 5 2 FDA PRO .. 7 PRO CONCEPTUAL FRAMEWORK .. 7 CONTENT VALIDITY .. 8 PRO .. 9 3 PRO .. 11 .. 11 PRO .. 12 PRO .. 12 PRO .. 13 .. 20 4 PRO .. 23 .. 23 .. 24.

2 25 .. 26 .. 28 .. 29 1) PRO .. 29 2) .. 32 3) 34 4) PRO .. 36 5) PRO .. 36 6) PRO .. 38 7) .. 38 8) .. 40 .. 43 .. 46 1 Patient Reported Outcome PRO HR-QOL Health related quality of life

3 2015 7 2015 8 7 PRO PRO PRO measure PRO ePRO electronic PRO PRO PRO PRO PRO 1 PRO 2 FDA PRO 3 PRO PRO PRO PRO 4 PRO PRO [1, 2]

4 PRO 2 1 PRO PRO PRO QOL Quality of Life QOL QOL [3] QOL QOL Health Related QOL, HR-QOL QOL 1948 WHO [4] PRO PRO QOL FDA [5] PRO PRO 2001 2 PRO [6]

5 PRO PRO 3 PRO 1- 1 3 1- 1 SF-36 EQ-5D VA S MPQ PainDETECT womac ODI JOABPEQ BPI EORTC-QLQ FA C T-G PSQI ESS BASDAI ACR core set DAS28 COPD SGRQ AQLQ HADS BDI SDISS SF-36 : MOS(Medical Outcome Study) 36-item Short-Form health survey, EQ-5D : EuroQol 5 Dimension, VAS : Visual Analog Scale, MPQ : McGill Pain Questionnaire, womac : Western Ontario & McMaster Universities osteoarthritis index , ODI : Oswestry Disability index , JOABPEQ : the Japanese Orthopaedic Association Back Pain Evaluation Questionnaire, BPI : Brief Pain Inventory, EORTC-QLQ.

6 The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, FAC T-G : Functional Assessment of Cancer-general, PSQI : Pittsburgh Sleep Quality index , ESS : Epworth Sleepiness Scale, BASDAI : Bath Ankylosing Spondylitis Disease Activity index , ACR core set : American College of Rheumatology core set DAS28 : Disease Activity Score 28, SGRQ : St George s Respiratory Questionnaire, AQLQ : Asthma Quality of Life Questionnaire, HADS : Hospital Anxiety and Depression scale, BDI : Beck Depression Inventory, SDISS : Sheehan Disability Scale Electronic PRO [ePRO] ePRO PRO site PRO eDiary ePRO [7] PRO 4 [6] 1.

7 PRO 2. caregiver 3. 4. FEV1 HbA1c 3,4 4 4 1 PRO ICH-E9 QOL [8] PRO 22 1 26 12 5 208 24 PRO 1- 2 24 7 6 8 PRO 1- 2 PRO 1- 2 PRO * ** PRO 112,116, 119 7 (24) VA S 821 2 (2) 239 2 (8) SF-NDI.

8 Short Form of the Nepean Dyspepsia index 259 1 (2) 634 1 (26) A 399 6 (46) HAQ : Health Assessment Questionnaire VA S 121 1 (3) VRS : Verbal Rating Scale 114 1 (1) 625 1 (14) 449 1 (2) Total four-symptom score 131 1 (5) * 87 3 ** 208 5 2007 2013 [9] 30,012 1887 PRO FDA 2009 PRO Guidance for Industry Patient-Reported Outcome Measures[5]

9 PRO PRO 2 PRO PRO FDA Comparative Effectiveness PRO FDA-DIA stat workshop [10] PRO Critical Path institute C-PAT H PRO [11] EMA PRO 2005 Reflection Paper on the Regulatory Guidance for the use of health related Quality of Life [3] 2009 Qualification of novel methodologies for drug development [12] Guideline on the evaluation of anticancer medicinal products in man [13]

10 2012 PRO Appendix 2016 11 The use of patient-reported outcome (PRO) measures in oncology studies [14] PRO 3 17 [15] QOL PRO 6 Patient-focused Drug Development [16] PRO 7 2 FDA PRO 2009 12 FDA FDA Guidance for Industry PRO Measures Use in Medical Product Development to Support Labeling Cl